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Abstract:
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There are tremendous unmet medical needs to develop therapies for rare diseases. Yet, conducting clinical trials in rare diseases face numerous challenges, ranging from low disease prevalence, lack of good understanding of disease natural history, lack of well-established endpoints, patient population heterogeneity, and other feasibility and scientific issues. The roundtable would propose to discuss statistical considerations on the design and analysis of clinical trials in rare disease areas, including trial designs that maximize use of patient information, more adaptive or flexible trial designs, trial designs that utilizes information from natural history, registry data, covariates, and/or disease modeling, choice of information rich or more sensitive endpoints, considerations on alternative endpoints including surrogates and composites, and the possibility of higher than conventionally accepted type I and type II error rates. The round table session will also discuss practical considerations on enrollment challenges, how to leverage from cross-discipline collaboration, and current thinking from regulatory agencies.
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